Pulmonary function generally remains stable in Duchenne until loss of ambulation. What is the trigger that occurs at loss of ambulation that initiates the cascade of pulmonary dysfunction?
What is the exact mechanism affecting the cardiopulmonary cascade of events that occurs in Duchenne?
What are the protective genetic modifiers that may be associated with a Duchenne phenotype more resistant to the development of cardiac and pulmonary fibrosis and subsequent pulmonary/cardiac dysfunction? . Are there genetic modifiers that may ameliorate or enhance the onset of cardiac and/or pulmonary dysfunction?
Is there a correlation between the development of abdominal/diaphragmatic fibrosis and the development of cardio-pulmonary dysfunction? Are there mechanisms (i.e., pulmonary excursion therapy) that may prevent/postpone the development of diaphragmatic fibrosis and subsequent pulmonary dysfunction?